TW202136516A - Iva型黏多醣病之治療 - Google Patents

Iva型黏多醣病之治療 Download PDF

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Publication number
TW202136516A
TW202136516A TW110103306A TW110103306A TW202136516A TW 202136516 A TW202136516 A TW 202136516A TW 110103306 A TW110103306 A TW 110103306A TW 110103306 A TW110103306 A TW 110103306A TW 202136516 A TW202136516 A TW 202136516A
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Taiwan
Prior art keywords
nucleotide sequence
seq
promoter
hgalns
raav
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TW110103306A
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English (en)
Chinese (zh)
Inventor
迪文 麥道加
梅勒錫 蘇巴 卡魯木錫
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美商銳進科斯生物股份有限公司
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Publication of TW202136516A publication Critical patent/TW202136516A/zh

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    • CCHEMISTRY; METALLURGY
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/06Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06004N-Acetylgalactosamine-6-sulfatase (3.1.6.4)
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/075Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0306Animal model for genetic diseases
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/33Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/22Vectors comprising a coding region that has been codon optimised for expression in a respective host
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/15Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Plant Substances (AREA)
  • Polysaccharides And Polysaccharide Derivatives (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
TW110103306A 2020-01-29 2021-01-28 Iva型黏多醣病之治療 TW202136516A (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202062967499P 2020-01-29 2020-01-29
US62/967,499 2020-01-29

Publications (1)

Publication Number Publication Date
TW202136516A true TW202136516A (zh) 2021-10-01

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ID=74673384

Family Applications (1)

Application Number Title Priority Date Filing Date
TW110103306A TW202136516A (zh) 2020-01-29 2021-01-28 Iva型黏多醣病之治療

Country Status (11)

Country Link
US (1) US20230064077A1 (enExample)
EP (1) EP4097238A1 (enExample)
JP (1) JP2023512043A (enExample)
KR (1) KR20220133900A (enExample)
AR (1) AR121201A1 (enExample)
AU (1) AU2021212749A1 (enExample)
BR (1) BR112022015036A2 (enExample)
CA (1) CA3168251A1 (enExample)
IL (1) IL294638A (enExample)
TW (1) TW202136516A (enExample)
WO (1) WO2021154956A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202039857A (zh) 2018-07-27 2020-11-01 美商銳進科斯生物股份有限公司 黏多醣病iva之治療

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2369002A1 (en) 1999-08-09 2011-09-28 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
SG10202108118RA (en) 2001-11-13 2021-08-30 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
ES2975413T3 (es) 2001-12-17 2024-07-05 Univ Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas
HUE054805T2 (hu) 2003-09-30 2021-09-28 Univ Pennsylvania Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik
EP3085389A1 (en) 2005-04-07 2016-10-26 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
WO2006132118A1 (ja) 2005-06-09 2006-12-14 Matsushita Electric Industrial Co., Ltd. 振幅誤差補償装置及び直交度誤差補償装置
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US8628966B2 (en) 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
CN107828820B (zh) 2010-10-27 2022-06-07 学校法人自治医科大学 用于向神经系统细胞导入基因的腺相关病毒粒子
US9409953B2 (en) 2011-02-10 2016-08-09 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
CN107012171B (zh) 2011-04-22 2021-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
EP3147295B2 (en) 2011-08-24 2023-11-22 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
EP3470523A1 (en) 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
CA2905952A1 (en) 2013-03-13 2014-10-02 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
PT3024498T (pt) 2013-07-22 2020-03-06 Childrens Hospital Philadelphia Variante aav e composições, métodos e usos para transferência genética em células, órgãos e tecidos
EP3564379A1 (en) 2013-09-13 2019-11-06 California Institute of Technology Selective recovery
NZ718926A (en) 2013-10-11 2021-12-24 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
US10746742B2 (en) 2014-04-25 2020-08-18 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
US12146169B2 (en) * 2018-05-30 2024-11-19 Esteve Pharmaceuticals, S.A. Adenoassociated virus vectors for the treatment of mucopolysaccharidoses type IV A
TW202039857A (zh) * 2018-07-27 2020-11-01 美商銳進科斯生物股份有限公司 黏多醣病iva之治療

Also Published As

Publication number Publication date
KR20220133900A (ko) 2022-10-05
WO2021154956A1 (en) 2021-08-05
EP4097238A1 (en) 2022-12-07
JP2023512043A (ja) 2023-03-23
AR121201A1 (es) 2022-04-27
CA3168251A1 (en) 2021-08-05
IL294638A (en) 2022-09-01
US20230064077A1 (en) 2023-03-02
BR112022015036A2 (pt) 2022-10-11
AU2021212749A1 (en) 2022-08-18

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